- Despite advancements, improved access to orphan drugs still needed -
TORONTO, Feb. 28, 2013 /CNW/ - Last night at the Canadian Organization
for Rare Disorders (CORD) Rare Disease Day Awards Gala Celebration, a
dozen "rare heroes" were honoured for their contributions to the rare
disease community in Canada. Held on the eve of International Rare
Disease Day, the event brought together the Canadian rare disease
community as part of CORD's two-day Rare Disease Day Conference, with
the hopeful theme of "From Worst to First: Transforming the Rare
Disease Experience in Canada."
"Given the progress our community has made in the past year, we felt it
was the right time to recognize those individuals and groups that have
inspired, challenged, and forever changed what it means to live with a
rare disorder," said Durhane Wong-Rieger, president of CORD.
Heroes of rare disease praised for contributions
In January 2013, CORD asked the rare disease community to nominate
heroes in six Rarity Award categories, and then asked the community to
vote on the nominees. The winners of the CORD's first-ever Rarity
Awards and Rare Honour Awards were announced at the Gala:
Members of Parliament Kirsty Duncan and Joy Smith were recognized for
their strong political leadership.
David K. Lee from Health Canada received a standing ovation for his
"dogged determination and tireless commitment" to drafting the orphan
drug regulations and for ensuring they met "world class" standards.
In the category of Patient Leadership, the community recognized CORD for
its role as a "catalyst" in rare disease policy and creating a strong
Dr. Cheryl Greenberg, geneticist at the Manitoba Institute of Child
Health, received the top award for Health Professional Leadership.
Dr. Lorne Greene from B.C. Children's Hospital was recognized for his
contribution to Scientific Excellence.
The Rarity Award of Honour went to the Rare Disease Foundation, led by
CORD also surprised its president, Durhane Wong-Rieger, with an
Outstanding Achievement Award for Volunteerism, recognizing six years
of service. Five other individuals were recognized for their overall contributions
to rare diseases. These included Barry Katsof, on behalf of the
Canadian Association of PNH Patients, 12-year-old Jonathan Pitre, a
hero and inspiration nominated by DEBRA Canada, Dr. Johanna Rommens,
researcher at the Toronto Hospital for Sick Children, Maureen Sauve, on
behalf of the Scleroderma Society of Canada, and Dr. Robert
Schellenberg, who was nominated along with the Primary Immunodeficiency
Clinic team at St. Paul's Hospital in Vancouver.
Awards were presented to the winners by CORD Board members including
John Adams (Chair), Caroline Laughlin (Vice-Chair), Maureen Smith
(Secretary), Charlene Doucette and Jim Mundy. Additional Board members
include Stephen McElroy (Treasurer), Kelly Gorman and Ashraf Ghadban.
"These awards demonstrate that rare disease heroes can be anyone. It can
be a 12-year-old boy who, despite living with an incredibly painful
disease, works tirelessly to raise awareness and funds for all those
affected by it," said Wong-Rieger. "It can be one of Canada's leading
geneticists who, on Christmas Day, drove through a minus-30-degree
snowstorm to pick up a mom and her 10-year-old son from Lebanon
arriving in Winnipeg to start clinical trials for a bone disease that
had already left the boy unable to walk. These are our incredible
Improved access to orphan treatments urged
Despite major advancements made in the rare disease community, improved
funding and access to treatments are still sought by CORD and rare
disease advocates across the country.
In October 2012, Federal Health Minister Leona Aglukkaq announced the
first Orphan Drug Regulatory Framework to improve access to new
medicines for rare and neglected diseases. This announcement came
after years of advocacy by the rare disease community, nearly 30 years
after the USA had passed the 1983 Orphan Drug Act, and 12 years after
the EU passed similar legislation.
As an indication of the significance of orphan drug legislation, there
had been only 10 drugs approved for rare diseases in the decade prior.
Since the legislation, there have been more than 400 orphan drugs
approved. However, less than half of the orphan drugs approved by
Health Canada are available to Canadian patients. Provincial drug plans have been reluctant to fund drugs for small
patient populations, discouraging developers from bringing new rare
disease drugs to Canada - despite the inclusion of Canadian patients in
clinical trials. Additionally, federal incentives for research for rare
disease drugs continue to lag.
"We are all inspired to work toward the implementation of the Orphan
Drug regulations and to develop a universal program to ensure drugs are
available to all appropriate patients across Canada," says Wong-Rieger.
"What we are ultimately aiming for is a national Rare Disease Institute
that can bring together all components of rare disease research, care
and treatment and all stakeholders in the rare diseases community."
CORD is Canada's national network for organizations representing all
those with rare disorders. CORD provides a strong common voice to
advocate for health policy and a healthcare system that works for those
with rare disorders. CORD works with governments, researchers,
clinicians and industry to promote research, diagnosis, treatment and
services for all rare disorders in Canada.
SOURCE: Canadian Organization for Rare Disorders (CORD)
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